NEW HOPE RESEARCH FOUNDATION

The New Hope Research Foundation has been established to
coordinate, conduct, and financially support:

Research on gene therapy and the means for infusing or delivering gene vectors to
correct lysosomal enzyme deficiencies of the central nervous system,

Clinical studies intended to show safety and efficacy of gene therapy for the central
nervous system aspects of lysosomal storage diseases,

Regulatory applications required to initiate investigations of clinical studies and the
regulatory submissions required to allow patient access to proven therapies, and

Education and communication for patients and families on the clinical manifestations
and molecular basis of gangliosidosis and related diseases, on the mechanism of gene
therapy, and on the status of related research and clinical studies.

The New Hope Research Foundation is conducting medical research to find a genetic cure for lysosomal storage diseases that affect the central nervous system. The medical research is being conducted by world experts at academic and hospital research centers. The New Hope Research Foundation serves to identify, fund, and coordinate these research activities.

The New Hope Research Foundation is being led by individuals with both a keen understanding of the diseases under investigation and a history of successful research and development projects. The New Hope Research Foundation has identified, funded, and coordinated research activities at world leading centers for genetic engineering, biochemistry, and pre-clinical studies.

Medical research conducted by the New Hope Research Foundation has supported major advancements in gene therapy. Recent scientific publications have summarized some of these advancements:

Tropak, M. B. et al. Construction of a hybrid beta-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo. Molecular therapy. Methods & clinical development 3, 15057, doi:10.1038/mtm.2015.57 (2016).

Karumuthil-Melethil, S. et al. Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Aav for the Treatment of Tay-Sachs Disease. Human gene therapy, doi:10.1089/hum.2016.013 (2016).

Osmon, K. J. et al. Systemic Gene Transfer of a Hexosaminidase Variant Using a scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice. Human gene therapy, doi:10.1089/hum.2016.015 (2016).

Keimel, J. G. et al., Gene Transfer to the Central Nervous System for Treatment of GM2 Gangliosidosis, Institute for Engineering in Medicine Annual Meeting (2015)

Sinici, I. et al. In cellulo examination of a beta-alpha hybrid construct of beta-hexosaminidase A subunits, reported to interact with the GM2 activator protein and hydrolyze GM2 ganglioside. PLoS ONE 8, e57908, doi:10.1371/journal.pone.0057908 (2013).

Clarke, J. T. et al., 2011, An open-label Phase I/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay-Sachs or Sandhoff variants). Molecular genetics and metabolism 102, 6-12